A bone marrow or cord blood transplant is the only known cure for WAS at this time. A bone marrow or cord blood transplant begins with chemotherapy, with or without radiation, to destroy the diseased cells and marrow. The transplant replaces diseased blood-forming cells with healthy ones. The type of transplant used for WAS is an allogeneic transplant. This type of transplant uses healthy blood-forming cells from a family member, unrelated donor, or umbilical cord blood unit.
For an allogeneic transplant, a patient gets chemotherapy, with or without radiation, prior to transplant to prepare his or her body for the treatment. Then the replacement cells are infused into the patient’s blood stream. From there, the cells find their way into the bone marrow, where they start making healthy red blood cells, white blood cells, and platelets.
The entire process, from start of chemotherapy or radiation until hospital discharge, can last weeks to months followed by many months of recovery at home.
Understanding if transplant would help your child with WAS
Our patient services coordinators can answer your questions and provide support and education to help you navigate your transplant journey.
Because allogeneic transplant is the only known cure for WAS, it is best to have a transplant as soon as possible. This is because children with WAS are at risk for dying of severe infections before their transplant provides a working immune system. In addition, children who have already had severe infections may be too weak to tolerate a transplant.
For these reasons, doctors recommend that children with WAS be referred to a transplant doctor as soon as they are diagnosed.1 A transplant doctor who is an expert in WAS can explain the risks and benefits of transplant.
In general, a child has a better chance to be cured by a transplant if:
- The transplant is done soon after diagnosis
- A child has not had severe infections
1. Recommended Timing for Transplant Consultation. Guidelines developed jointly by National Marrow Donor Program/Be The Match and the American Society for Blood and Marrow Transplantation (ASBMT). Available at: marrow.org/md-guidelines